Splenomegaly

Ryvu Therapeutics to Present Preclinical Data on RVU120 and Synthetic Lethality Programs at the 2024 AACR Annual Meeting

Retrieved on: 
Wednesday, March 6, 2024

Ryvu's partner Menarini to present data on MEN1703 (SEL24), demonstrating promising anti-tumor activity in preclinical models of myelofibrosis both as a single agent and combined with ruxolitinib.

Key Points: 
  • Ryvu's partner Menarini to present data on MEN1703 (SEL24), demonstrating promising anti-tumor activity in preclinical models of myelofibrosis both as a single agent and combined with ruxolitinib.
  • "We are excited to present our latest preclinical data at the AACR Annual Meeting, showcasing our significant progress in advancing novel small molecule therapies for oncology.
  • This year, we will present data from our most advanced preclinical project on MTA-cooperative PRMT5 inhibitors, the lead program within Ryvu's synthetic lethality pipeline.
  • Ryvu has developed potentially best-in-class MTA-cooperative PRMT5 inhibitors showing favorable drug-like properties and effective PRMT5 inhibition dependent on MTA binding.

Drug Farm Receives Rare Pediatric Disease Designation from the U.S. FDA for DF-003 to Treat ROSAH Syndrome

Retrieved on: 
Saturday, January 13, 2024

Drug Farm , a clinical-stage biopharmaceutical company announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation for the company’s alpha-kinase 1 (ALPK1) inhibitor, DF-003, to treat patients with ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache) Syndrome.

Key Points: 
  • Drug Farm , a clinical-stage biopharmaceutical company announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation for the company’s alpha-kinase 1 (ALPK1) inhibitor, DF-003, to treat patients with ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache) Syndrome.
  • DF-003 is currently being evaluated in a Phase 1 trial ( NCT05997641 ) to assess safety and pharmacokinetics in normal healthy volunteers.
  • “Pediatric patients living with ROSAH Syndrome face a significant unmet need with limited options to treat vision loss,” said Jeysen Yogaratnam, Chief Medical Officer, Drug Farm.
  • “Obtaining Rare Pediatric Disease Designation recognizes the serious and debilitating complications of this rare disease and upholds our goal to provide DF-003 as the first targeted drug for potential treatment in patients afflicted with ROSAH Syndrome.”

Studies Highlight Both Novel Treatments and Enduring Value of Older Approaches

Retrieved on: 
Sunday, December 10, 2023

SAN DIEGO, Dec. 10, 2023 /PRNewswire/ -- Research findings being presented at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition highlight new treatment approaches that are enabling patients to live longer or better, judicious uses of older treatment approaches, and how a powerful, relatively new prognostic tool is helping identify patients who are more or less likely to benefit from both older and newer treatments.

Key Points: 
  • Two other studies presented in this session spotlight ways in which a novel prognostic tool is demonstrating its value across a range of blood cancers.
  • The researchers used MRD to determine how long patients in the targeted-agents group should continue treatment.
  • These cells develop into specialized blood cells, such as red and white blood cells and platelets, that also carry the mutation.
  • By comparison, progression-free survival (PFS) for patients receiving the standard of care was 76.8% with a death rate of 7%.

Drug Farm Doses First Patient in Part 3 of Phase 1 Chronic Hepatitis B Trial

Retrieved on: 
Tuesday, September 26, 2023

Drug Farm, a clinical-stage biopharmaceutical company, announced today that it has dosed the first chronic hepatitis B patient in Part 3 of its Phase 1 clinical trial (ANZCTR Trial ID: ACTRN12621000592842 ).

Key Points: 
  • Drug Farm, a clinical-stage biopharmaceutical company, announced today that it has dosed the first chronic hepatitis B patient in Part 3 of its Phase 1 clinical trial (ANZCTR Trial ID: ACTRN12621000592842 ).
  • This trial is evaluating Drug Farm’s first-in-class ALPK1 agonist, DF-006, as a monotherapy and in combination with standard-of-care nucleos(t)side reverse transcriptase inhibitors in treatment-naïve and virologically suppressed patients with chronic hepatitis B infection.
  • Drug Farm is a private biotechnology company developing innovative treatments targeting innate immunity for hepatitis B, heart and kidney diseases, and ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache) syndrome.
  • Using the IDInVivo platform, Drug Farm has identified novel innate immunity pathways and targets and is now rapidly advancing multiple first-in-class drug candidates into clinical development.

Drug Farm Doses First Patient in Phase 1 Trial for ALPK1 Inhibitor, DF-003

Retrieved on: 
Tuesday, September 19, 2023

Drug Farm, a clinical-stage biopharmaceutical company, announced today that the first subject in the Phase 1 clinical trial ( NCT05997641 ) evaluating DF-003 in healthy volunteers has been dosed.

Key Points: 
  • Drug Farm, a clinical-stage biopharmaceutical company, announced today that the first subject in the Phase 1 clinical trial ( NCT05997641 ) evaluating DF-003 in healthy volunteers has been dosed.
  • A gain-of-function mutation in ALPK1 also causes a rare genetic disease, ROSAH syndrome, that leads to blindness.
  • “The Phase 1 study will evaluate DF-003 in healthy volunteers with single and multiple ascending doses that will inform dose selection for the Phase 2 programs.
  • Using the IDInVivo platform, Drug Farm has identified novel innate immunity pathways and targets and is now rapidly advancing multiple first-in-class drug candidates into clinical development.

Ojjaara (momelotinib) approved in the US as the first and only treatment indicated for myelofibrosis patients with anemia

Retrieved on: 
Saturday, September 16, 2023

GSK plc (LSE/NYSE: GSK) today announced that the US Food and Drug Administration (FDA) has approved Ojjaara (momelotinib) for the treatment of intermediate or high-risk myelofibrosis, including primary myelofibrosis or secondary myelofibrosis (post-polycythemia vera and post-essential thrombocythemia), in adults with anemia.

Key Points: 
  • GSK plc (LSE/NYSE: GSK) today announced that the US Food and Drug Administration (FDA) has approved Ojjaara (momelotinib) for the treatment of intermediate or high-risk myelofibrosis, including primary myelofibrosis or secondary myelofibrosis (post-polycythemia vera and post-essential thrombocythemia), in adults with anemia.
  • Ojjaara is a once-a-day, oral JAK1/JAK2 and activin A receptor type 1 (ACVR1) inhibitor.
  • Given this high unmet need, we are proud to add Ojjaara to our oncology portfolio and address a significant medical need in the community.
  • About 40% of patients have moderate to severe anemia at the time of diagnosis, and nearly all patients are estimated to develop anemia over the course of the disease.7,8,9,10 Physicians have had limited treatment options to treat myelofibrosis patients with anemia.

Ryvu Therapeutics Reports 2023 Half-Year Financial Results and Provides Corporate Update

Retrieved on: 
Wednesday, September 13, 2023

Updated clinical and preclinical data on RVU120 were presented at the European Hematology Associated (EHA) Congress in June 2023.

Key Points: 
  • Updated clinical and preclinical data on RVU120 were presented at the European Hematology Associated (EHA) Congress in June 2023.
  • $14M in non-dilutive grant funding was secured from the Medical Research Agency (ABM) – the largest grant obtained to date by Ryvu.
  • KRAKOW, Poland, Sept. 13, 2023 (GLOBE NEWSWIRE) -- Ryvu Therapeutics (WSE: RVU), a clinical-stage drug discovery and development company focusing on novel small molecule therapies that address emerging targets in oncology, reported today financial results for the first half of 2023 and provided a corporate update.
  • “The last months of 2023 were a productive period for Ryvu as we made significant developments across our clinical programs, collaboration activity, and strengthening our balance sheet”, said Pawel Przewiezlikowski, CEO of Ryvu Therapeutics.

MediciNova Receives a Notice of Intention to Grant for a New Patent Covering MN-001 and MN-002 for the Treatment of Advanced NASH in Europe

Retrieved on: 
Tuesday, August 15, 2023

Once issued, the patent maturing from this allowed patent application is expected to expire no earlier than May 2035.

Key Points: 
  • Once issued, the patent maturing from this allowed patent application is expected to expire no earlier than May 2035.
  • The allowed claims cover MN-001 (tipelukast) or MN-002 for the treatment of a patient diagnosed with advanced NASH.
  • In addition, the allowed claims cover MN-001 (tipelukast) or MN-002 for reducing hepatic fibrosis and hepatic scarring in a patient with advanced NASH.
  • The U.S. Patent and Trademark Office previously granted a patent which covers MN-001 and MN-002 for advanced NASH with fibrosis and similar patents were granted in Japan, Korea, and Canada.”

NS-018, an Investigational Treatment for Myelofibrosis, Receives Orphan Drug Designation from the European Commission

Retrieved on: 
Tuesday, August 8, 2023

PARAMUS, N.J., Aug. 8, 2023 /PRNewswire/ -- NS Pharma, Inc. announced today that the European Commission (EC) has granted Orphan Drug Designation to NS-018 (ilginatinib) an oral, selective JAK2 inhibitor which is being investigated for the treatment of myelofibrosis (MF).

Key Points: 
  • NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru Nakai)
    PARAMUS, N.J., Aug. 8, 2023 /PRNewswire/ -- NS Pharma, Inc. announced today that the European Commission (EC) has granted Orphan Drug Designation to NS-018 (ilginatinib) an oral, selective JAK2 inhibitor which is being investigated for the treatment of myelofibrosis (MF).
  • The EC Orphan Drug Designation is issued to investigational treatments for diseases that affect fewer than 5 in 10,000 people in the European Union and are life-threatening or chronically debilitating.
  • The designation provides for a ten-year marketing exclusivity period.
  • In the US, NS-018 received Orphan Drug Designation by the U.S. Food and Drug Administration in December 2022.